The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials
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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials

According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…

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A Drug That Can Mimic Exercise May Be Closer Than You Thought
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A Drug That Can Mimic Exercise May Be Closer Than You Thought

Scientists in California at Salk Institute for Biological Studies are currently developing a drug that can mimic the effects in the body usually obtained from exercise, reports the National Post.…

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A CRISPR Gene Editing Technique Could Fix Many of the Mutations Responsible For Duchenne Muscular Dystrophy
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A CRISPR Gene Editing Technique Could Fix Many of the Mutations Responsible For Duchenne Muscular Dystrophy

CRISPR gene editing technology is still mostly in its infancy, but the new tool is already causing a lot of excitement, particularly in the treatment of genetically-linked diseases. A recent…

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Spinraza, a New Drug For SMA Patients Might Be Available Sooner Than Later in the UK
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Spinraza, a New Drug For SMA Patients Might Be Available Sooner Than Later in the UK

There is new hope for patients and their families suffering from spinal muscular atrophy (SMA) reported AOL UK News. SMA is a physically debilitating and aggressive disease, and now a new…

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Researchers Close to Medical Breakthroughs Concerning  Amyotrophic Lateral Sclerosis and Muscular Dystrophy
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Researchers Close to Medical Breakthroughs Concerning Amyotrophic Lateral Sclerosis and Muscular Dystrophy

Originally reported by Columbia Daily Tribune, researchers from the University of Missouri have made huge strides towards treating, and maybe even curing, two fatal diseases: muscular dystrophy and Amyotrophic Lateral…

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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy
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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy

Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…

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PTC Therapeutics Continue To Fight For Duchenne Muscular Dystrophy Community
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PTC Therapeutics Continue To Fight For Duchenne Muscular Dystrophy Community

Even after the third denial from the U.S. FDA, Food and Drug Administration, PTC Therapeutics Incorporation will not stop fighting for the drug Translarna that they believe will help patients…

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Turning Skin Cells Into Muscle Cells: Treatment for Muscular Disorders
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Turning Skin Cells Into Muscle Cells: Treatment for Muscular Disorders

There is potential for a new treatment for Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness.To learn more about this…

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